In vivo gene editing to halt the clock before it’s too late
A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.

In vivo gene editing to halt the clock before it’s too late
A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.
Locus-specific editing of the 21-hydroxylase gene restores adrenal function in a CAH model
Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
Tangram seeks clinical trial clearance for TGM-312 for MASH
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Next-gen therapeutic elastase selectively kills cancer cells
A paper from Onchilles Pharma Inc. and collaborating institutions details the development of N-17350, a next-generation therapeutic elastase optimized for intratumoral delivery that targets the neutrophil elastase pathway.
CTX-10726 bispecific antibody shows robust preclinical efficacy
Researchers from Compass Therapeutics Inc. detailed the preclinical characterization of CTX-10726, a bispecific, tetravalent antibody that simultaneously targets VEGF-A and PD-1.
Nikang Therapeutics patents new CDK2 and CDK4 degradation inducers
Nikang Therapeutics Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a cyclin-dependent kinase 2 (CDK2)- and/or CDK4-targeting moiety through a linker reported to be useful for the treatment of cancer.
CT-202, a tumor-selective pH-dependent Nectin-4/CD3 TCE
Nectin-4 is a cell-adhesion molecule that is highly expressed in several malignancies, including bladder, colorectal, lung and breast cancers, while exhibiting minimal expression in most normal adult tissues.

Conferences

Opko’s OPK-88006 shows hepatoprotection in MASH
American Association for the Study of Liver Diseases
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH) and obesity.
Gene therapies aim for the big goal of edits in vivo
Endocrine/metabolic
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications...
Selective CDK2 inhibitor is active in multiple solid tumor models
New compound
Researchers from Eilean Therapeutics LLC and collaborators presented the discovery and characterization of a new, selective CDK2 inhibitor showing potent in vitro and in vivo activity at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics.
Small-molecule SMARCA2 inhibitors to treat SMARCA4-mutant cancers
New compound
At the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, researchers from Onco3r Therapeutics BV presented a novel series of selective SMARCA2 small-molecule inhibitors with a best-in-class potency and selectivity profile.

Cancer

Bakuchiol derivatives against breast cancer
Bakuchiol, a phenolic meroterpenoid from the seeds of Psoralea corylifolia L., has shown promise as an antitumor drug, so researchers at Bengbu Medical University synthesized novel derivatives of bakuchiol bearing modifications on the aromatic ring.

Infection

Harmane-based quaternary ammonium salt against drug-resistant S. aureus
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms.

Invivyd nominates VBY-329 as candidate for RSV prevention
Immune
Invivyd Inc. has nominated VBY-329 as a preclinical development candidate for the prevention of respiratory syncytial virus (RSV) among neonates, infants and children.
In lethal influenza, repair the lung before it’s too late
NIH researchers report that in severe influenza, survival improves at late stages only when antivirals are combined with therapies that repair lung damage or limit harmful T-cell responses, explaining why anti-inflammatory treatments alone are often ineffective.

Neurology/psychiatric

Gemma’s GB-221 gains trial clearance in Brazil for SMA1
Regulatory
Gemma Biotherapeutics Inc.’s GB-221, a novel gene therapy for spinal muscular atrophy type 1 (SMA1), has received clinical trial clearance from ANVISA, Brazil’s health regulatory agency.
RFK skirts promise as CDC alters vaccine-autism webpage
Regulatory
Changes to a U.S. CDC website regarding autism and vaccines has sparked a backlash from numerous scientific and other groups, placing HHS Secretary Robert F. Kennedy Jr. (RFK) in the spotlight once again for appearing to break promises made earlier this year to secure his nomination.
CSPC’s SYH-2056 cleared to enter clinic in China for depression
Regulatory
CSPC Pharmaceutical Group Ltd. has obtained approval from China’s National Medical Products Administration (NMPA) to initiate clinical trials with the company’s selective 5-HT2A receptor agonist, SYH-2056 tablets, for the treatment of depression.
Takeda divulges new OX2R agonists
Patents
Takeda Pharmaceutical Co. Ltd. has synthesized heterocyclic compounds acting as orexin OX2 receptor (OX2R; HCRTR2) agonists reported to be useful for the treatment of narcolepsy, among others.
MJFF grant backs Scineuro’s SNP-614 for Parkinson’s disease
Collaboration
Scineuro Pharmaceuticals Holdings Ltd. has received a $5 million research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to accelerate the preclinical development of the company’s novel LRRK2-targeted antisense oligonucleotide (ASO) program, SNP-614, for Parkinson’s disease.

Endocrine/metabolic

UK Biobank delivers largest metabolomics dataset of 500K profiles
Science
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.

Biomarkers

Mesothelin is biomarker, potential target in arthritic bone damage
Musculoskeletal
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...

With transdiagnostic approach, smaller target groups aren’t inevitably smaller markets
Neurology/psychiatric
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the...
Anchoring, and bootstrapping, psychiatry into the precision medicine era
Neurology/psychiatric
Psychiatry has struggled to enter the precision medicine era. But through a mix of innovations and bootstrapping, progress is coming to the field. Scientists are working on improving diagnoses by investigating potential biomarkers and collection methods.

Gastrointestinal

UK study reveals inconsistencies in global microbiome research
Regulatory
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical patents new compounds for MASH
Patents
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex patents new NOD2 activators for Crohn’s disease
Patents
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.
Numab and Kaken sign agreement for NM-81 for IBD
Collaboration
Numab Therapeutics AG and Kaken Pharmaceutical Co. Ltd. have entered into a strategic licensing and co-development agreement for NM-81, a multi-specific antibody for the treatment of inflammatory bowel disease (IBD).
Korro Bio nominates KRRO-121 as development candidate
Endocrine/metabolic
Korro Bio Inc. has nominated KRRO-121 as its next development candidate, for the treatment of patients with hyperammonemia, including patients with urea cycle disorders and hepatic encephalopathy.
Genfit and Everzom form research collaboration in liver failure
Collaboration
Genfit SA has established a research collaboration with Everzom SAS to expand its acute-on-chronic liver failure (ACLF) research via exosome-based regenerative technology. The partners plan to conduct exploratory studies to assess efficacy of...

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